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HQK-1001

National Institutes of Health

Papers overview

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2016
2016
of the short-chain fatty acid derivative 2,2-dimethylbutyrate (HQK-1001) in inducing Hb F in 76 patients with SCD. [5] The… Expand
2014
2014
This placebo‐controlled phase II study evaluated the pharmacodynamics, efficacy and safety of 2,2‐dimethylbutyrate (HQK‐1001), a… Expand
2014
2014
To the editor: β-thalassemia syndromes comprise a global health burden. Hemoglobin E (HbE)–β thalassemia represents 60% of… Expand
2014
2014
The β-thalassaemia syndromes represent a World Health Organization-designated global health burden (Weatherall et al, 2010… Expand
2013
2013
2,2‐dimethylbutyrate (HQK‐1001), an orally‐bioavailable promoter‐targeted fetal globin gene‐inducing agent, was evaluated in an… Expand
2013
2013
β‐thalassaemia intermedia (BTI) syndromes cause haemolytic anaemia, ineffective erythropoiesis, and widespread complications… Expand
2012
2012
Therapeutics which reduce the pathology in sickle cell syndromes are needed, particularly noncytotoxic therapeutics. Fetal… Expand
2010
2010
Abstract 943 Definitive therapies to reduce the underlying pathology in sickle cell syndromes are still needed, particularly non… Expand
2009
2009
Abstract 977 Several classes of HbF inducers, including chemotherapeutic agents, ESAs, and short chain fatty acids (SCFADs) have… Expand
2008
2008
Development of non-cytotoxic, orally-active therapeutics, which induce fetal globin production, would be beneficial for treatment… Expand